CN110636865A - 修饰的寡核苷酸及其治疗用途 - Google Patents

修饰的寡核苷酸及其治疗用途 Download PDF

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Publication number
CN110636865A
CN110636865A CN201880026265.5A CN201880026265A CN110636865A CN 110636865 A CN110636865 A CN 110636865A CN 201880026265 A CN201880026265 A CN 201880026265A CN 110636865 A CN110636865 A CN 110636865A
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CN
China
Prior art keywords
compound
protein
pharmaceutical composition
moiety
weight
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
CN201880026265.5A
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English (en)
Chinese (zh)
Inventor
亚历山大·罗洛夫
N·C·詹内斯基
C·E·考尔曼
M·P·汤普森
P·A·伯廷
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Vybyl Inc
University of California San Diego UCSD
Original Assignee
Vybyl Inc
University of California San Diego UCSD
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Application filed by Vybyl Inc, University of California San Diego UCSD filed Critical Vybyl Inc
Publication of CN110636865A publication Critical patent/CN110636865A/zh
Pending legal-status Critical Current

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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/68—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
    • A61K47/6801—Drug-antibody or immunoglobulin conjugates defined by the pharmacologically or therapeutically active agent
    • A61K47/6803—Drugs conjugated to an antibody or immunoglobulin, e.g. cisplatin-antibody conjugates
    • A61K47/6807—Drugs conjugated to an antibody or immunoglobulin, e.g. cisplatin-antibody conjugates the drug or compound being a sugar, nucleoside, nucleotide, nucleic acid, e.g. RNA antisense
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/30—Macromolecular organic or inorganic compounds, e.g. inorganic polyphosphates
    • A61K47/42—Proteins; Polypeptides; Degradation products thereof; Derivatives thereof, e.g. albumin, gelatin or zein
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/542—Carboxylic acids, e.g. a fatty acid or an amino acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/62—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being a protein, peptide or polyamino acid
    • A61K47/64—Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
    • A61K47/643—Albumins, e.g. HSA, BSA, ovalbumin or a Keyhole Limpet Hemocyanin [KHL]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Inorganic Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Dermatology (AREA)
  • Immunology (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Genetics & Genomics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
CN201880026265.5A 2017-03-22 2018-03-21 修饰的寡核苷酸及其治疗用途 Pending CN110636865A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762475185P 2017-03-22 2017-03-22
US62/475,185 2017-03-22
PCT/US2018/023578 WO2018175592A1 (en) 2017-03-22 2018-03-21 Modified oligonucleotides and therapeutic uses thereof

Publications (1)

Publication Number Publication Date
CN110636865A true CN110636865A (zh) 2019-12-31

Family

ID=63586170

Family Applications (1)

Application Number Title Priority Date Filing Date
CN201880026265.5A Pending CN110636865A (zh) 2017-03-22 2018-03-21 修饰的寡核苷酸及其治疗用途

Country Status (9)

Country Link
US (1) US20200046846A1 (enExample)
EP (1) EP3600439A4 (enExample)
JP (1) JP2020514383A (enExample)
KR (1) KR20190123351A (enExample)
CN (1) CN110636865A (enExample)
AU (1) AU2018237139A1 (enExample)
CA (1) CA3057292A1 (enExample)
SG (1) SG11201908771YA (enExample)
WO (1) WO2018175592A1 (enExample)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN116133691A (zh) * 2020-09-16 2023-05-16 阿斯利康(瑞典)有限公司 与脂肪酸缀合的寡核苷酸

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN117980003A (zh) * 2021-09-10 2024-05-03 嘉德治疗有限责任公司 核酸的脂肪酸缀合物
WO2025028656A1 (ja) * 2023-08-02 2025-02-06 日東電工株式会社 オリゴヌクレオチドを製造する方法

Citations (3)

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WO2007112414A2 (en) * 2006-03-27 2007-10-04 Isis Pharmaceuticals, Inc. Conjugated double strand compositions for use in gene modulation
EP2716758A1 (en) * 2011-06-03 2014-04-09 National University Corporation Hokkaido University OLIGONUCLEOTIDE DERIVATIVE, OLIGONUCLEOTIDE DERIVATIVE-CONTAINING PHARMACEUTICAL COMPOSITION FOR TREATMENT AND PHARMACEUTICAL COMPOSITION FOR DIAGNOSIS, AND OLIGONUCLEOTIDE DERIVATIVE FOR REGULATION OF miRNA FUNCTION
WO2016149501A2 (en) * 2015-03-18 2016-09-22 The California Institute For Biomedical Research Modified therapeutic agents and compositions thereof

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US8969543B2 (en) * 2003-04-03 2015-03-03 Bioneer Corporation SiRNA-hydrophilic polymer conjugates for intracellular delivery of siRNA and method thereof
KR101224828B1 (ko) * 2009-05-14 2013-01-22 (주)바이오니아 ｓｉＲＮＡ 접합체 및 그 제조방법
GB0910723D0 (en) * 2009-06-22 2009-08-05 Sylentis Sau Novel drugs for inhibition of gene expression
RU2599449C1 (ru) * 2011-12-15 2016-10-10 Байонир Корпорейшн Новые конъюгаты олигонуклеотидов и их применение
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AU2014364589B2 (en) * 2013-12-18 2020-02-27 The California Institute For Biomedical Research Modified therapeutic agents, stapled peptide lipid conjugates, and compositions thereof
JP6773677B2 (ja) * 2015-03-17 2020-10-21 アローヘッド ファーマシューティカルズ インコーポレイテッド 改善されたジスルフィド含有アルキン連結剤
WO2017030973A1 (en) * 2015-08-14 2017-02-23 University Of Massachusetts Bioactive conjugates for oligonucleotide delivery
AU2016326392B2 (en) * 2015-09-22 2021-02-11 The Regents Of The University Of California Modified cytotoxins and their therapeutic use

Patent Citations (3)

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WO2007112414A2 (en) * 2006-03-27 2007-10-04 Isis Pharmaceuticals, Inc. Conjugated double strand compositions for use in gene modulation
EP2716758A1 (en) * 2011-06-03 2014-04-09 National University Corporation Hokkaido University OLIGONUCLEOTIDE DERIVATIVE, OLIGONUCLEOTIDE DERIVATIVE-CONTAINING PHARMACEUTICAL COMPOSITION FOR TREATMENT AND PHARMACEUTICAL COMPOSITION FOR DIAGNOSIS, AND OLIGONUCLEOTIDE DERIVATIVE FOR REGULATION OF miRNA FUNCTION
WO2016149501A2 (en) * 2015-03-18 2016-09-22 The California Institute For Biomedical Research Modified therapeutic agents and compositions thereof

Non-Patent Citations (3)

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Title
ARNEDO A ET AL.: "Albumin nanoparticles as carriers for a phosphodiester oligonucleotide", 《INTERNATIONAL JOURNAL OF PHARMACEUTICS》 *
ILYA DOVYDENKO ET AL.: "Method of carrier-free delivery of therapeutic RNA importable into human mitochondria:Lipophilic conjugates with cleavable bonds", 《BIOMATERIALS》 *
XIN MING ET AL.: "Albumin-based nanoconjugates for targeted delivery of therapeutic oligonucleotides", 《BIOMATERIALS》 *

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN116133691A (zh) * 2020-09-16 2023-05-16 阿斯利康(瑞典)有限公司 与脂肪酸缀合的寡核苷酸

Also Published As

Publication number Publication date
AU2018237139A1 (en) 2019-10-17
EP3600439A1 (en) 2020-02-05
KR20190123351A (ko) 2019-10-31
JP2020514383A (ja) 2020-05-21
SG11201908771YA (en) 2019-10-30
WO2018175592A1 (en) 2018-09-27
US20200046846A1 (en) 2020-02-13
EP3600439A4 (en) 2021-01-13
CA3057292A1 (en) 2018-09-27

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